US Pharm. 2014;39(10):54.

Through the FDA Safety and Innovation Act and the Prescription Drug User Fee Act, the FDA is expanding its efforts to expedite the development and review of promising therapies for serious conditions using fast-track, breakthrough-therapy, accelerated approval, and priority-review designations. In these cases, the FDA may engage in more presubmission communications with sponsors and review applications in 6 months, enabling the drug to be made available once it is determined that benefits outweigh risks. Orphan-drug and fee-waiver designations provide financial incentives to drug companies, but do not affect the review process.

Trends in Approvals: In 2013, the FDA approved 27 new molecular entities (NMEs). Of these, 11% were designated breakthrough therapy; 33%, 37%, and 7% were designated first-in-class, priority review, and accelerated approval, respectively. Although from 2005 to 2013 the number of NME applications did not consistently increase, a mean of 25.2 NMEs were approved annually, despite wide fluctuations (from 18 in 2007 to 39 in 2012). The number of NME applications ranged from 23 (2010) to 41 (2012), and from 52% (2005) to 95.1% (2012) of them were approved each year, yielding a mean annual approval rate of 74%. A high number of NME applications were approved in 2010 (91.3%) and 2012 (95.1%).

Fast Track: There was a significant decrease in the mean number of meetings per active investigational New Drug Application and per new drug or biologic license application (NDA/BLA)—from 0.29 and 2.49, respectively, in 2009 to 0.26 and 2.33 in 2012. The proportion of meetings denied dropped from 14.2% in 2009 to 6.7% in 2011; the number of meetings scheduled but not held increased from 69.3 (2009) to 72.5 (2011). In 2011, the FDA reviewed 89% of 91 fast-track action requests; 79% were granted status within the statutory 60-day period (median 51 days).

First Cycle: There were more approvals for drugs with priority-review applications than for nonpriority drugs (62% vs. 34%). Of 77 NDA/BLAs submitted, 47% were approved during the first cycle of the review process, and subsequent multicycle review resulted in approval of 23% of applications; orphan-designated drugs had approval rates of 33% and 12%, respectively. Of 46 products with end-of-phase 2 (EOP2) meetings, 52% received first-cycle approval versus 29% of products without EOP2 meetings. Larger U.S.-based sponsors were likelier than small, inexperienced drug developers to gain first-cycle approval (64% vs. 33%). The first-cycle approval rate for sponsors with prior FDA-approved drugs was 51%, versus 30% for sponsors with no prior approved drugs. Eighty percent of drugs with first-cycle approval had postmarketing commitments.

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